AAT + Corticosteroids for Graft-versus-Host Disease

Recruiting at 25 trial locations

Overseen By Trial Registration Coordinator

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: CSL Behring

Must be taking: Corticosteroids

Must not be taking: AAT, other GVHD drugs

Disqualifiers: Relapsed malignancy, Chronic GVHD, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

136 Participants Needed

Trial Summary

What is the purpose of this trial?

Study CSL964_5001 will investigate the efficacy of AAT with corticosteroids compared with corticosteroids alone as first line therapy for patients with high-risk acute GVHD

Research Team

Study Director

Principal Investigator

CSL Behring

Eligibility Criteria

This trial is for patients aged 12 or older who have acute GVHD after a stem cell transplant and need systemic therapy with corticosteroids. It's not for those previously treated with AAT, having cancer that's returned or not responding to treatment, using steroids within the last week before GVHD onset, or currently on other drugs for GVHD.

Inclusion Criteria

I am 12 years old or older.

I have had a transplant from any donor or undergone any intensity of preparation treatment.

I have acute GVHD after a stem cell transplant.

Exclusion Criteria

My cancer has come back or is getting worse.

I have chronic GVHD or overlap syndrome that started before or at the time of joining.

I started taking systemic corticosteroids within 7 days before my acute GVHD symptoms began.

Treatment Details

Interventions

• Alpha-1 antitrypsin (AAT)
• Placebo

Trial Overview The study compares two treatments for acute GVHD: one group receives Alpha-1 antitrypsin (AAT) plus corticosteroids, while the other gets a placebo alongside corticosteroids. The goal is to see if adding AAT improves outcomes.

Participant Groups

Group I: AAT Experimental Treatment 1 Intervention

Alpha-1 antitrypsin (AAT) is a lyophilized powder for intravenous administration

Group II: Placebo Placebo Group 1 Intervention

Albumin solution administered intravenously

Alpha-1 antitrypsin (AAT) is already approved in United States, European Union for the following indications:

Approved in United States as Alpha-1 antitrypsin for:

• Chronic obstructive pulmonary disease (COPD) associated with alpha-1 antitrypsin deficiency

Approved in European Union as Alpha-1 antitrypsin for:

• Chronic obstructive pulmonary disease (COPD) associated with alpha-1 antitrypsin deficiency

Who Is Running the Clinical Trial?

CSL Behring

Lead Sponsor

Trials

204

Recruited

1,207,000+

Dr. Paul McKenzie

CSL Behring

Chief Executive Officer since 2023

PhD in Chemical Engineering from Carnegie Mellon University, B.S. in Chemical Engineering from the University of Pennsylvania

Dr. Bill Mezzanotte

CSL Behring

Chief Medical Officer since 2021

MD from Duke University

National Institutes of Health (NIH)

Collaborator

Trials

2,896

Recruited

8,053,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Blood and Marrow Transplant Clinical Trials Network

Collaborator

Trials

51

Recruited

14,600+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials

3,987

Recruited

47,860,000+